Clinical Trial Update: Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C [Phase 1, Phase 2]

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Summary

Phase 1/2 trial evaluating intravenous VTS-270 for infantile liver disease in Niemann-Pick Disease, Type C has completed and results have been posted.

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Diseases / indications

• Niemann-pick Disease Type

Drugs mentioned

• VTS-270 (2-hydroxypropyl-beta-cyclodextrin)

Snippet

Sponsor: Washington University School of Medicine | Conditions: Niemann-Pick Disease, Type C | Phase 1, Phase 2 | Status: COMPLETED | Results posted: 2025-12-12